CRISPR by Designโ„ข

Alameda, CA
Joined April 2017
Announcing our first clinical trial. Scribe has secured regulatory clearance from Australiaโ€™s @TGAgovau to initiate a first-in-human clinical study of STX-1150 for the treatment of hypercholesterolemia, a major driver of atherosclerotic cardiovascular disease (ASCVD). Details๐Ÿงต
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Next week, weโ€™re in Hamburg, Germany, for #ESGCT2026! Principal Scientist Jason Fernandes (@jdf_ev) will present data on our epigenetic silencing technology ELXR and our engineering approach for improving potency and specificity. ELXR underpins STX-1150, our clinical-stage PCSK9-targeting therapy designed to down-regulate PCSK9 transcription and deliver ultra-long-acting, potent LDL-C reductions for years โ€“ without permanently altering DNA. Swing by on Tuesday to learn more about our epigenetic silencers at @ESGCT Congress. More details below. ๐Ÿงฌ Next generation CasX-based epigenetic silencers engineered for potency, specificity, and durability: from high-throughput discovery to clinical translation ๐Ÿชง Session 2b: (Epi)genome editing I ๐Ÿ“… Tuesday, October 27, 2026 at 6:15 pm CET #ESGCTHamburg2026
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At Ilmac Lausanne, our co-founder and VP External Innovation Brett Staahl gave a keynote on a belief we hold firmly at Scribe: AI is only useful if it is trained on the right datasets. Brettโ€™s talk, โ€œPurpose-built CRISPR genetic medicines โ€” Leveraging human know-how and AI to create better therapeutics faster,โ€ showcased how Scribeโ€™s CRISPR by Designโ„ข iterative molecular engineering approach engineers CRISPR systems into precision medicines AND learns from the biology, turning those data into better predictions for Scribeโ€™s platform technologies, XE and ELXR, and future therapeutic programs. The goal is to use AI and machine learning to move from broad screening toward targeted prediction, so we can test more hypotheses better, faster. He explained that while purpose-built CRISPR genetic medicines are the output, the proprietary sequence-function knowledge base is the compounding asset. Brett also joined the โ€œAI & Quantum in Life Sciencesโ€ panel discussion that clarified actionable steps that biopharma companies can take to implement AI/ML and reimagine their workflows. Watch his interview to learn more about our work at Scribe ๐Ÿ‘‡ youtube.com/watch?v=IeQ9R_0dโ€ฆ
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Next week, our CEO @BenjaminLOakes will speak at the @Biocom CEO Summit in Sonoma. Industry leaders can tune in for the โ€œCEO Perspectives: Pharma BD&Lโ€ session โ€“ a real-world tutorial for biotech CEOs and executive teams โ€“ on Friday, October 23. This #BiocomCEOSummit panel will walk through how business development deals actually move through biotech and pharmaceutical organizations, breaking down the practical mechanics, workflows, and decision points that shape whether a deal gets done or stalls along the way.
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Welcome to lesson #5 of ๐—ฆ๐—ฐ๐—ฟ๐—ถ๐—ฏ๐—ฒ ๐—ฆ๐˜†๐—น๐—น๐—ฎ๐—ฏ๐˜‚๐˜€, our educational series exploring how genetic medicine can tackle some of healthโ€™s most critical challenges. Weโ€™ve started to explore how CRISPR works and how its main components comprise a tool that can be reprogrammed to target almost any sequence in the genome. Now weโ€™ll dive further into the artful process of how CRISPR actually finds and edits DNA. Leave your questions down in the comments! ๐Ÿ“– ๐—–๐—ฅ๐—œ๐—ฆ๐—ฃ๐—ฅ'๐˜€ ๐—บ๐—ผ๐—น๐—ฒ๐—ฐ๐˜‚๐—น๐—ฎ๐—ฟ ๐—บ๐—ฎ๐—ฐ๐—ต๐—ถ๐—ป๐—ฒ๐—ฟ๐˜† ๐˜‚๐—ฝ ๐—ฐ๐—น๐—ผ๐˜€๐—ฒ: ๐—ฃ๐—ฎ๐—ฟ๐˜ ๐Ÿฎ Letโ€™s return to two important components that make up the CRISPR system: the guide RNA (gRNA) and the Cas enzyme. In our last lesson, we learned that the gRNA (the โ€œGPS systemโ€) leads the Cas enzyme (โ€œthe scissorsโ€) to the targeted DNA sequence. This week, we will explore how these CRISPR components are delivered to cells, assembled, transported into the nucleus, and the process by which they scan the genome to make a defined edit at the target DNA sequence. Dive in with our Substack post and follow along in our video. ๐Ÿ”— scribetx.substack.com/p/scriโ€ฆ
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Weโ€™re proud to have the support of @CIRMnews in advancing our cardiovascular programs into the clinic and to patients who need them most. CIRM features these two therapies โ€“ STX-1200 for Lp(a)-driven atherosclerotic cardiovascular disease and STX-1400 for severe hypertriglyceridemia โ€“ and our recent IPO in a new blog post. โ€œThis investment helps Scribe to advance two programs that aim to deliver practical, one-time therapeutic solutions to patients throughout California that will enable them to avoid a lifetime of healthcare friction spent managing disease imperfectly.โ€ - Brett Staahl, Scribe co-founder and Vice President of External Innovation More from CIRM: blog.cirm.ca.gov/research-anโ€ฆ
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Congratulations to Ioana L. Aanei, our Director of Business Development and Alliance Management, for being honored in @biospace's 40 Under 40! Hereโ€™s what makes her one of biotechโ€™s most exemplary leaders: โญ Drives Scribeโ€™s strategic partnerships to advance engineered CRISPR-based genetic medicines for heart disease and more โญ Community builder dedicated to connecting cross-functional resources within the biotech ecosystem to better serve patients and the broader field โญ Co-founded Calamores, an organization focused on parental well-being and science-backed self-care โญ Sustained advocate for emerging talent and active mentor through @Biocom and the @UCBerkeley Big Ideas program Read more here: biospace.com/biospace-40-undโ€ฆ Huge thanks to BioSpace for recognizing our Scribe team two years in a row. ๐Ÿ’ช
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In Phoenix for the @alliancerm's Cell & Gene Meeting on the Mesa? Donโ€™t miss todayโ€™s innovation spotlight presentation by our CFO David Parrot at 5:15 pm MST. Heโ€™ll discuss our work engineering purpose-built in vivo genetic medicines for highly prevalent conditions like cardiometabolic disease. #CGMesa26
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Welcome to lesson #4 of ๐—ฆ๐—ฐ๐—ฟ๐—ถ๐—ฏ๐—ฒ ๐—ฆ๐˜†๐—น๐—น๐—ฎ๐—ฏ๐˜‚๐˜€, our educational series exploring how genetic medicine can tackle some of healthโ€™s most critical challenges. Now that weโ€™ve covered CRISPRโ€™s surprising and unassuming origin story, weโ€™ll boil down how the technology actually works โ€“ and itโ€™s much easier to understand than you might expect. Leave any questions down in the comments! ๐Ÿ“– ๐—›๐—ผ๐˜„ ๐—–๐—ฅ๐—œ๐—ฆ๐—ฃ๐—ฅ ๐—ช๐—ผ๐—ฟ๐—ธ๐˜€: ๐—ฃ๐—ฎ๐—ฟ๐˜ ๐Ÿญ It may seem complex, but the beauty of CRISPR is that itโ€™s a remarkably elegant tool. That simplicity is why scientists around the world keep finding new uses for it beyond its original purpose as an immune defense system. In our last Syllabus lesson, we learned that CRISPR harnesses biological functions from bacteria that allow them to fight viruses. The entire CRISPR system has two main components: the ๐—–๐—ฎ๐˜€ ๐—ฒ๐—ป๐˜‡๐˜†๐—บ๐—ฒ and the ๐—ด๐˜‚๐—ถ๐—ฑ๐—ฒ ๐—ฅ๐—ก๐—” (โ€œ๐—ด๐—ฅ๐—ก๐—”โ€). CRISPRโ€™s revolutionary function is its ability to recognize, attach to and edit pieces of our DNA that predispose us to disease. In order to do this, it needs to identify the problematic sequence of DNA, and then cut it so the cellโ€™s own repair machinery can modify it. Thatโ€™s where the gRNA and Cas come in. The gRNA finds the target DNA, and the Cas enzyme cuts it. Read all about it in our full post on Substack, and stay tuned for a deeper dive in part 2! ๐Ÿ”— scribetx.substack.com/p/scriโ€ฆ
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Itโ€™s #WorldHeartDay and #CholesterolEducationMonth. Take action against cardiovascular disease for yourself and your loved ones by knowing the numbers that make it the leading cause of death ๐Ÿ‘‡ โค๏ธโ€๐Ÿฉน Every 40 seconds, someone in the US has a heart attack. โค๏ธโ€๐Ÿฉน Americans have more than 1.5 million heart attacks and strokes each year. โค๏ธโ€๐Ÿฉน About 1 in 5 heart attacks are silent. โค๏ธโ€๐Ÿฉน 63 million US adults have high LDL cholesterol (LDL-C), known as โ€œbad cholesterol.โ€ โค๏ธโ€๐Ÿฉน LDL-C is one of the largest drivers of heart disease, but only 20% of US patients with high LDL-C get treated. To protect your #HeartHealth, get your LDL-C checked and treated early. Recent studies show: ๐Ÿ’ก People at lower risk of heart disease may gain the most from lowering LDL-C. ๐Ÿ’ก The benefits build over time: Keeping LDL-C lower for longer could mean fewer procedures down the line. Sources: @American_Heart, @CDCgov, European Journal of Preventive Cardiology, American Journal of Preventive Cardiology Learn about our work at Scribe to develop CRISPR-based genetic medicines that are safe and potent enough to address high LDL-C โ€“ among other major risk factors of cardiometabolic disease โ€“ to improve heart health for all: scribetx.com
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Heart disease at a glance: heart.org/-/media/PHD-Files-โ€ฆ Separating cholesterol facts from myths: cdc.gov/cholesterol/about/myโ€ฆ More on World Heart Day: world-heart-federation.org/wโ€ฆ
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This week, tune into two events where our VP of Research Sarah Denny will break down the science and engineering approach behind our CRISPR-based genetic medicines. 1๏ธโƒฃ 7th Genome Editing Therapeutics Summit hosted by the Cell & Gene Therapy Event Series, AKA #crispr2.0 (September 28-30, Boston) 2๏ธโƒฃ Tech Spotlight: AI and CRISPR Genome Editing Webinar hosted by @Tech_Networks (September 29, virtual)
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At #IPAVision 2026, our co-founder and CEO @BenjaminLOakes took the stage to discuss epigenetic silencing and gene editing as some of todayโ€™s most promising technologies and how they can address the worldโ€™s leading cause of death โ€“ cardiovascular disease. Our thanks to the Institute for Portfolio Alternatives for the invitation to lead the conversation on breakthrough technologies for medicine, AI, and more.
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In @biospace, learn about our differentiated epigenetic silencing technology, ELXR, and clinical-stage lead candidate STX-1150, purpose-built to offer durable cardiovascular risk reduction to patients in need. STX-1150 is designed to silence PCSK9 and deliver sustained reductions of LDL-C or โ€œbadโ€ cholesterol after a single dose, without permanently changing the underlying DNA sequence. Read more from Gabrielle Masson: biospace.com/business/scribeโ€ฆ Learn more about ELXR: scribetx.substack.com/p/engiโ€ฆ
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Welcome to lesson #3 of ๐—ฆ๐—ฐ๐—ฟ๐—ถ๐—ฏ๐—ฒ ๐—ฆ๐˜†๐—น๐—น๐—ฎ๐—ฏ๐˜‚๐˜€, our educational series exploring how genetic medicine can tackle some of healthโ€™s most critical challenges. We previously covered the potential of genetic medicine to restore and improve everyone's health. In this post, weโ€™ll go back in time to understand where CRISPR โ€“ one of the most powerful technologies for genetic medicine โ€“ came from and how itโ€™s being used to develop breakthrough therapies today. Have a question or a topic we should cover in a future post? Drop it in the comments. ๐Ÿ“– ๐—›๐—ผ๐˜„ ๐—–๐—ฅ๐—œ๐—ฆ๐—ฃ๐—ฅ ๐˜„๐—ฎ๐˜€ ๐—ฑ๐—ถ๐˜€๐—ฐ๐—ผ๐˜ƒ๐—ฒ๐—ฟ๐—ฒ๐—ฑ Decades before CRISPR was harnessed into a gene editing technology, scientists understood that DNA holds the natural blueprint for disease prevention. The problem was turning that knowledge into safe, reliable treatments. They lacked a tool precise enough to make efficient, accurate edits to the human genome. The discovery of CRISPR closed that gap, and it all started with bacteria. For the full post, head over to our Substack where we cover: โ€ข The origin of CRISPR as a natural defense for bacteria to fend off viruses โ€ข How Jennifer Doudna (one of Scribeโ€™s co-founders) and Emmanuelle Charpentier showed that CRISPR could be engineered and reprogrammed to target any piece of DNA, not just viral DNA โ€ข How CRISPR is being used today to improve human health across the globe ๐Ÿ”— scribetx.substack.com/p/scriโ€ฆ โœ๏ธ Image credit: IGI Glossary Icon Collection by Christine Liu of Two Photon Art for the Innovative Genomics Institute
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Hear directly from our CEO @BenjaminLOakes about Scribeโ€™s lead asset STX-1150, a clinical-stage epigenetic silencing therapy purpose-built to repress PCSK9, lower LDL cholesterol, and offer durable cardiovascular risk reduction to patients in need. More on our cardiometabolic pipeline, engineering approach compared to first-generation CRISPR technologies, and more in his conversation with @bradloncar. Full video is up on @BiotechTV: biotechtv.com/post/scribe-thโ€ฆ
๐‡๐‚๐– ๐Ÿ๐ŸŽ๐Ÿ๐Ÿ”: @scribetx which represents the next generation of CRISPR, had its IPO in July - the first clinical program targets PCSK9. With Co-Founder & CEO @BenjaminLOakes. $SCTX Full video: biotechtv.com/post/scribe-thโ€ฆ
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๐Ÿงฌ๐Ÿ” We built two-factor authentication into gene silencing. Read about it in our new blog and paper โ€“ links in the comments. We call this approach ๐—ฆ๐—ฒ๐—พ๐˜‚๐—ฒ๐—ป๐˜๐—ถ๐—ฎ๐—น ๐—ฃ๐—ฟ๐—ผ๐—ผ๐—ณ๐—ฟ๐—ฒ๐—ฎ๐—ฑ๐—ถ๐—ป๐—ด. Itโ€™s engineered into ELXR, the epigenetic silencing technology built on our CRISPR-CasX platform. The results? ๐—จ๐—น๐˜๐—ฟ๐—ฎ-๐—น๐—ผ๐—ป๐—ด ๐—ด๐—ฒ๐—ป๐—ฒ ๐˜€๐—ถ๐—น๐—ฒ๐—ป๐—ฐ๐—ถ๐—ป๐—ด ๐˜„๐—ถ๐˜๐—ต๐—ผ๐˜‚๐˜ ๐—ฐ๐—ต๐—ฎ๐—ป๐—ด๐—ถ๐—ป๐—ด ๐—ฎ๐—ป๐˜† ๐——๐—ก๐—”. โ†’ More than 10-fold greater specificity โ†’ More than 4-fold greater silencing activity across every target tested โ†’ No toxicity in stress tests, rescuing growth defects observed with Cas9-based silencers โ†’ Mechanism mirrors the cellโ€™s natural regulation Learn more about this work led by Emeric J Charles, Christie C Sze, @BenjaminLOakes, Sarah Denny, @jdf_ev, and team below.
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This week, donโ€™t miss these upcoming talks by our co-founders: 1๏ธโƒฃ Fireside chat with CEO @BenjaminLOakes at the @HCWCO 28th Annual Global Investment Conference. This will take place Tuesday, September 15 at 2:30 p.m. ET in New York. #HCWConference 2๏ธโƒฃ Panel discussion where VP of External Innovation @BStaahl will discuss โ€œAdvances in Gene Editingโ€ at the Cell & Gene Therapy Symposium. The session begins Thursday, September 17 at 11:00 a.m. PT in La Jolla and is hosted by the @CALifeSciences, @CIRMnews, and Gene Therapy Initiative at @UCSanDiego.
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๐—ง๐—ต๐—ฒ ๐—™๐˜‚๐˜๐˜‚๐—ฟ๐—ถ๐˜€๐˜: ๐—ง๐—ฒ๐—ฐ๐—ต๐—ป๐—ผ๐—น๐—ผ๐—ด๐—ถ๐—ฒ๐˜€ ๐—ง๐—ต๐—ฎ๐˜ ๐—–๐—ผ๐˜‚๐—น๐—ฑ ๐—ฅ๐—ฒ๐—ฑ๐—ฒ๐—ณ๐—ถ๐—ป๐—ฒ ๐—›๐˜‚๐—บ๐—ฎ๐—ป๐—ถ๐˜๐˜† โ€” ๐—ผ๐—ฟ ๐—ก๐—ฒ๐˜ƒ๐—ฒ๐—ฟ ๐—™๐˜‚๐—น๐—น๐˜† ๐——๐—ฒ๐—น๐—ถ๐˜ƒ๐—ฒ๐—ฟ ๐˜๐˜ถ๐˜ด๐˜ช๐˜ฐ๐˜ฏ, ๐˜ข๐˜ฅ๐˜ท๐˜ข๐˜ฏ๐˜ค๐˜ฆ๐˜ฅ ๐˜ฏ๐˜ถ๐˜ค๐˜ญ๐˜ฆ๐˜ข๐˜ณ, ๐˜ˆ๐˜, ๐˜ฒ๐˜ถ๐˜ข๐˜ฏ๐˜ต๐˜ถ๐˜ฎ ๐˜ต๐˜ฆ๐˜ค๐˜ฉ๐˜ฏ๐˜ฐ๐˜ญ๐˜ฐ๐˜จ๐˜ช๐˜ฆ๐˜ด, ๐˜ฃ๐˜ช๐˜ฐ๐˜ฎ๐˜ฆ๐˜ฅ๐˜ช๐˜ค๐˜ช๐˜ฏ๐˜ฆ, ๐˜ข๐˜ฏ๐˜ฅ ๐˜จ๐˜ฆ๐˜ฏ๐˜ฆ๐˜ต๐˜ช๐˜ค ๐˜ฆ๐˜ฏ๐˜จ๐˜ช๐˜ฏ๐˜ฆ๐˜ฆ๐˜ณ๐˜ช๐˜ฏ๐˜จ Next week, our co-founder and CEO @BenjaminLOakes will discuss todayโ€™s most ambitious and promising technologies โ€“ including gene editing and silencing โ€“ at #IPAVision 2026, the Institute for Portfolio Alternativesโ€™ premier event. Panel details below. ๐Ÿ“… Wednesday, September 16, 2026 ๐Ÿ•— 5:15-6:05 p.m. ET ๐Ÿ“ Atlanta
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